4.7 Article

Precision oncology medicines and the need for real world evidence acceptance in health technology assessment: Importance of patient involvement in sustainable healthcare

相关参考文献

注意:仅列出部分参考文献,下载原文获取全部文献信息。
Article Oncology

Defining the role of real-world data in cancer clinical research: The position of the European Organisation for Research and Treatment of Cancer

Robbe Saesen et al.

Summary: The precision medicine paradigm in oncology has sparked interest in integrating real-world data (RWD) into cancer research. RWD studies tend to focus on collecting and analyzing observational data, but randomized controlled trials (RCTs) have the potential to generate strong evidence. The European Organisation for Research and Treatment of Cancer (EORTC) prioritizes pragmatic trials and trials-within-cohorts to generate robust RWD, but will consider observational research based on the target trial principle if random allocation is not feasible. New EORTC-sponsored trials may also include concurrent prospective cohorts.

EUROPEAN JOURNAL OF CANCER (2023)

Review Pharmacology & Pharmacy

Real-World Data in the Postapproval Setting as Applied by the EMA and the US FDA

Sarah Mofid et al.

Summary: This study analyzes the regulatory decisions in postmarketing surveillance activities that suggest the use of real-world data (RWD) approved by the European Medicines Agency (EMA) and the US Food and Drug Administration (FDA). The findings show an increasing use of RWD to support regulatory decision-making, with registries being the most common source of data. The study highlights the potential of RWD to inform regulatory decisions on clinical interventions' benefits and risks.

CLINICAL THERAPEUTICS (2022)

Article Oncology

Market access to new anticancer medicines for children and adolescents with cancer in Europe

Reineke A. Schoot et al.

Summary: This study reveals significant variability in HTA decision making for pediatric anticancer medicines across nine European countries, highlighting the need for collaboration and alignment to facilitate robust scientific assessments, taking into account methodological challenges in pediatric oncology.

EUROPEAN JOURNAL OF CANCER (2022)

Article Biochemistry & Molecular Biology

Delivering precision oncology to patients with cancer

Joaquin Mateo et al.

Summary: Precision medicine is reshaping cancer care, but accessibility to all patients remains a challenge. This Perspective highlights the importance of addressing the challenges in implementing precision oncology, including equal access to genomics tests, robust evidence for new drugs and technologies, interpretation of genomics data by physicians, and empowering patients in shared decision-making.

NATURE MEDICINE (2022)

Review Genetics & Heredity

Clinical trial design in the era of precision medicine

Elena Fountzilas et al.

Summary: Recent breakthroughs in biotechnology have allowed for the identification of unique molecular features that drive malignancies. Precision medicine has used next-generation sequencing and matched targeted therapy/immunotherapy to transform the outlook for fatal cancers. Multiple trial designs and personalized combination treatment strategies have been implemented to accelerate drug evaluation and individualized treatment.

GENOME MEDICINE (2022)

Editorial Material Oncology

A global approach to long-term follow-up of targeted and immune-based therapy in childhood and adolescence

Mark W. Kieran et al.

Summary: Efforts have been made to understand the long-term toxicities of traditional cancer treatments in children, but there is still a lack of knowledge regarding the health impact of novel targeted and immunotherapy agents. ACCELERATE, a multi-stakeholder collaboration, has initiated the development of an international long-term follow-up data registry to address this gap and provide critical safety data to support regulatory requirements and guide physicians and families in survivorship planning.

PEDIATRIC BLOOD & CANCER (2021)

Review Oncology

Precision oncology: a clinical and patient perspective

Ulrik N. Lassen et al.

Summary: Precision oncology provides individualized treatment for each patient based on the unique DNA fingerprint of their cancer, with advancements in DNA sequencing technologies and decision-making tools improving therapy matching and outcomes, while increasing hope for the future. This approach has shifted cancer treatment strategies towards targeting actionable mutations found across multiple cancer types, and has proven success in improving outcomes, quality of life, and overcoming drug resistance and relapse mechanisms. Addressing challenges in its use will further improve therapy matching to patients.

FUTURE ONCOLOGY (2021)

Review Pharmacology & Pharmacy

Use of Real-world Data for New Drug Applications and Line Extensions

Winona R. Bolislis et al.

CLINICAL THERAPEUTICS (2020)

Article Health Care Sciences & Services

Methods for Population-Adjusted Indirect Comparisons in Health Technology Appraisal

David M. Phillippo et al.

MEDICAL DECISION MAKING (2018)

Article Medical Informatics

Improving Patient Involvement in Medicines Research and Development:: A Practical Roadmap

Jan Geissler et al.

THERAPEUTIC INNOVATION & REGULATORY SCIENCE (2017)

Review Genetics & Heredity

Systematic review on the evaluation criteria of orphan medicines in Central and Eastern European countries

Tamas Zelei et al.

ORPHANET JOURNAL OF RARE DISEASES (2016)

Article Medicine, General & Internal

Increasing value and reducing waste in research design, conduct, and analysis

John P. A. Ioannidis et al.

LANCET (2014)